BioMarin provides highlights of 4 Years of clinical data from ongoing phase 1/II study of valoctocogene roxaparvovec gene therapy for severe hemophilia A.

BioMarin Pharmaceutical Inc. announced an update to its previously reported results of an open-label Phase 1/II study of valoctocogene roxaparvovec, an investigational gene therapy treatment for adults with severe hemophilia A. The data have been submitted as a late-breaking abstract to the World Federation of Hemophilia (WFH) Virtual Summit to be held June 14-19, 2020.

The four-year update for the 6e13 vg/kg and three-year update for the 4e13 vg/kg cohorts demonstrated that all subjects in both cohorts remain off prophylactic Factor VIII treatment since receiving their single dose of valoctocogene roxaparvovec. Cumulative mean annualized bleed rates (ABR) remain less than one (1) in both cohorts and below pre-treatment baseline levels. The mean ABR in year four for the 6e13 vg/kg cohort was 1.3, and the mean ABR in year three for the 4e13 vg/kg cohort was 0.5. Over the past year, six of the seven participants in the 6e13 vg/kg cohort and five of the six participants in the 4e13 vg/kg cohort remain free of spontaneous bleeds . Factor VIII activity levels declined commensurate with the most recent years’ observations and remain in a range to provide hemostatic efficacy.